Nicole W.
- Country
- The Netherlands
- Department
- Child Neurology
Research Overview
- Long‑Term Monitoring of Insertional Oncogenesis–Related Malignancy Risk in Early‑Onset Metachromatic Leukodystrophy Patients Treated with Atidarsagene Autotemcel (OTL‑200)
- ION356-CS1: A Phase 1b Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Patients with Pelizaeus-Merzbacher Disease
- Evaluation of Deferiprone's Efficacy on Gross Motor Function in Pediatric Pelizaeus-Merzbacher Disease: A Proof-of-Concept Clinical Trial
Data & Insights
Has run more than one trial at 1 of 10 partner sites.
Specializations
Neurological Disorders
This investigator specializes in rare neurological conditions, with particular emphasis on Pelizaeus-Merzbacher disease (PMD) and related disorders affecting the central nervous system.
- Leukodystrophies
- Myelin disorders
- Genetic neurological conditions
The research focuses on evaluating novel therapeutic approaches for managing rare neurological disorders affecting myelin development.
Therapeutic Development
The investigator studies innovative treatments for rare neurological conditions, including deferiprone and targeted molecular therapies like ION356 for genetic disorders.
- Motor function assessment
- Pharmacological interventions
- Treatment safety evaluation
Research activities encompass the evaluation of novel therapeutic compounds for rare neurological conditions.
Adult-Onset Disorders
The investigator conducts research in adult-onset leukoencephalopathy, particularly focusing on conditions with axonal spheroids and pigmented glia (ALSP).
- White matter disorders
- Axonal pathologies
- Progressive neurological conditions
The research extends to investigating targeted treatments such as iluzanebart for adult-onset neurological disorders.
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